Ionis Pharmaceuticals Inc - Company Profile
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Business Description
Ionis Pharmaceuticals Inc. (Ionis), formerly known as ISIS Pharmaceuticals Inc., is a biotechnology company dedicated to discovering and developing antisense medicines to treat a wide range of serious diseases. Its therapies are built on proprietary, patented antisense technology, which is designed to target disease-related RNA and reduce the production of harmful proteins. Ionis runs its business through one reportable segment, Ionis Operations, and concentrates on building a pipeline across multiple therapeutic areas, including oncology, cardiovascular and metabolic disorders, pulmonary conditions, neurological diseases, and infectious diseases.
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R&D Overview
Ionis conducts research and development to advance its pipeline and preclinical candidates of antisense medicines for the treatment of a broad range of diseases. Its research is centered on disorders linked to RNA targets present in specific tissues. Key pipeline programs include Eplontersen for transthyretin amyloidosis (ATTR), Olezarsen for FCS and severe hypertriglyceridemia (SHTG), Donidalorsen for hereditary angioedema (HAE), and Zilganersen for Alexander disease. Its partnership portfolio includes Ulefnersen for ALS, Tofersen for SOD1-ALS, Pelacarsen for elevated lipoprotein(a) (Lp(a)) and cardiovascular disease, and Bepirovirsen for chronic hepatitis B virus (HBV). In FY2025, the company spent US$915.6 million on R&D, which increased 1.5%YoY. Ionis also holds patents covering chemically modified nucleosides and oligonucleotides, drug design motifs, ligand-conjugated antisense (LICA) technology, therapeutic methods, oligonucleotide sequences, and drug compositions.
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